Acute Myeloid Leukaemia is a malignant blood disorder primarily affecting adults, with low survival rates and various risk factors including age and genetic predisposition. Treatment combines chemotherapy with targeted drugs, while ongoing research focuses on uncovering molecular mechanisms for personalized therapies. This FAQ explores the advancements in Acute Myeloid Leukaemia treatment, focusing on innovative drugs, ongoing clinical trials, and their potential impact on patient outcomes.

1. How does the incidence of Acute Myeloid Leukaemia vary globally, and what factors contribute to these variations?

The global incidence of Acute Myeloid Leukaemia varies due to factors such as genetic predispositions, environmental exposures, and specific risk factors, including genetic mutations like FLT3, NPM1, and CEBPA. These mutations drive the need for precise genetic profiling to tailor therapies for better outcomes. Acute Myeloid Leukaemia affects around 150,000 individuals globally. Asia, particularly Mainland China, leads in incidence due to factors like the prevalence of Hepatitis B. Europe follows, with higher incidences reported in countries like Germany and France. North America, primarily the US and Canada, also contribute significantly. Egypt and South Africa contribute moderately to the rest of the world.

2. What are the recent breakthroughs and upcoming treatment options being investigated for Acute Myeloid Leukaemia?

Advancements in precision medicine for Acute Myeloid Leukaemia involve personalized treatments guided by molecular profiles, including targeted therapies such as FLT3, IDH1, and IDH2 inhibitors, along with drugs like Venetoclax and Gemtuzumab Ozogamicin. Approved drugs like Midostaurin, Gilteritinib, and Venetoclax target specific mutations, offering effective options for relapsed or refractory Acute Myeloid Leukaemia cases. Investigational immunotherapies like BiTE antibodies and CAR-T Cell therapy show promise, targeting CD33 and CD123. These targeted therapies and immunotherapies help in better understanding of Acute Myeloid Leukaemia mutations, making a shift towards precision medicine for better management of the disease.

3. What are the main categories of approved precision oncology drugs for treating Acute Myeloid Leukaemia and what specific mutations do they target?

The approved precision oncology drugs for Acute Myeloid Leukaemia can be classified into many types based on their mechanism of action and target mutations. These categories include FLT3 kinase inhibitors (which target FLT3 mutations), IDH1 inhibitors (for IDH1 mutations), IDH2 inhibitors (for IDH2 mutations), anti-CD33 antibody-drug conjugates (for CD33-positive Acute Myeloid Leukaemia), BCL-2 inhibitors (for different Acute Myeloid Leukaemia subtypes), and hypomethylating agents (commonly used along with targeted therapies). Each drug within these categories is designed to specifically target and inhibit certain genetic mutations or pathways involved in the development and progression of Acute Myeloid Leukaemia.

4. What are the recent FDA-approved drugs for Acute Myeloid Leukaemia?

In the past two years, several drugs have received US FDA approval for Acute Myeloid Leukaemia treatment. Ivosidenib, approved in May 2022, is indicated for use in combination with Azacitidine to treat newly diagnosed Acute Myeloid Leukaemia with a susceptible IDH1 mutation. Olutasidenib capsules, approved in December 2022, are specifically used for relapsed or refractory Acute Myeloid Leukaemia cases with a susceptible IDH1 mutation. Quizartinib, approved in July 2023, is indicated for the treatment of newly diagnosed FLT3 ITD-positive Acute Myeloid Leukaemia in adults. Additionally, the LeukoStrat CDx FLT3 Mutation Assay received approval as a companion diagnostic for Quizartinib.

5. What is the global landscape of Acute Myeloid Leukaemia clinical trials, and how do recruitment rates vary across regions?

Since 2019, the global biotech and biopharma industry initiated over 1,000 clinical trials for Acute Myeloid Leukaemia. North America takes the lead, followed by the Asia-Pacific, Europe, and the rest of the world. The United States dominates in North America, while Mainland China and Australia represent a significant portion of trials in the Asia-Pacific. Spain and France take the lead in Acute Myeloid Leukaemia clinical trials in Europe and Israel leads in the rest of the world. Moreover, clinical trials in the Asia-Pacific region showed shorter recruitment durations and faster patient recruitment rates in comparison to the US and Europe possibly due to their larger and more diverse patient population.